自体造血干细胞移植大剂量免疫抑制治疗后多发性硬化症患者的生活质量

A. Polushin, E. Lopatina, A. Tsynchenko, Yury R. Zalyalov, I. Skiba, A.  V. Shegurova, N. Totolyan, A. D. Kulagin, T. Ionova, T. P. Nikitina, N. Porfirieva, A. A. Skoromets
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The single-center observational study included patients with MS (n = 21, mean age 35.5 ± 6.2 (28–50) years; men/women — 11/10; remitting MS (RMS) n = 16, secondary-progressive (SPMS) n = 4, primaryprogressive (PPMS) n = 1), after HDIT–AHSCT (cyclophosphamide-rituximab conditioning regimen). Assessment of the quality of life (SF-36, FAMS, CSP-MS42, HADS) and the degree of disability in all patients was carried out before and 12 months after HDIT–AHSCT.Results. 12 months after HDIT–AHSCT, more than 50% of patients showed signifi cant improvement in quality of life according to the scales of viability and social functioning of the SF-36 questionnaire, as well as according to the scales of general well-being and additional problems of the FAMS questionnaire. 57% of patients had a signifi cant improvement in the overall FAMS score. According to the CSP-MS42 questionnaire, a decrease in weakness (p < 0.05), feelings of constant fatigue and rapid fatigue during exercise was revealed. 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引用次数: 0

摘要

导言。多发性硬化症(MS)是一种慢性自身免疫性中枢神经系统(CNS)神经退行性疾病。高剂量免疫抑制治疗联合自体造血干细胞移植(HDIT-AHSCT)是治疗多发性硬化症最有效的方法,对提高患者生活质量和抑制残疾进展具有积极作用。本研究旨在分析接受 HDIT- AHSCT 方法的多发性硬化症患者在不同临床疗效组的生活质量动态。这项单中心观察性研究纳入了接受HDIT-AHSCT(环磷酰胺-利妥昔单抗调理方案)治疗的多发性硬化症患者(n=21,平均年龄35.5 ± 6.2 (28-50)岁;男性/女性-11/10;缓解型多发性硬化症(RMS)n=16,继发性进展型(SPMS)n=4,原发性进展型(PPMS)n=1)。所有患者的生活质量(SF-36、FAMS、CSP-MS42、HADS)和残疾程度在HDIT-AHSCT前和HDIT-AHSCT后12个月进行评估。HDIT-AHSCT治疗12个月后,根据SF-36问卷的生存能力和社会功能量表,以及FAMS问卷的一般幸福感和其他问题量表,50%以上的患者生活质量有明显改善。57% 的患者的 FAMS 总分有明显改善。根据 CSP-MS42 问卷调查,患者的乏力感(p < 0.05)、持续疲劳感和运动时的快速疲劳感均有所减轻。移植后,焦虑略有减少(p = 0.91),抑郁明显减少(p = 0.011)。预期的动态变化得到了证实:在临床症状改善的患者中,大部分生活质量指标得到了改善;在病情稳定的患者中,大部分生活质量指标在HDIT-AHSCT前后相似;在HDIT-AHSCT后临床症状恶化的患者中,生活质量指标恶化。这些数据表明,为了获得最佳临床效果,提高患者的生活质量,有必要进一步优化患者选择标准和 HDIT-AHSCT 的各个阶段。
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Quality of life of patients with multiple sclerosis after high-dose immunosuppressive therapy with autologous hematopoietic stem cell transplantation
Introduction. Multiple sclerosis (MS) is a chronic autoimmune and neurodegenerative disease of the central nervous system (CNS). High-dose immunosuppressive therapy with autologous hematopoietic stem cell transplantation (HDIT–AHSCT) is the most eff ective method of treating MS, which has a positive eff ect on the quality of life of patients and inhibition of the progression of disability.The aim of the study was to analyze the dynamics of quality of life in patients with MS who underwent the HDIT– AHSCT method in groups with diff erent clinical effi  cacy of this method.Material and methods. The single-center observational study included patients with MS (n = 21, mean age 35.5 ± 6.2 (28–50) years; men/women — 11/10; remitting MS (RMS) n = 16, secondary-progressive (SPMS) n = 4, primaryprogressive (PPMS) n = 1), after HDIT–AHSCT (cyclophosphamide-rituximab conditioning regimen). Assessment of the quality of life (SF-36, FAMS, CSP-MS42, HADS) and the degree of disability in all patients was carried out before and 12 months after HDIT–AHSCT.Results. 12 months after HDIT–AHSCT, more than 50% of patients showed signifi cant improvement in quality of life according to the scales of viability and social functioning of the SF-36 questionnaire, as well as according to the scales of general well-being and additional problems of the FAMS questionnaire. 57% of patients had a signifi cant improvement in the overall FAMS score. According to the CSP-MS42 questionnaire, a decrease in weakness (p < 0.05), feelings of constant fatigue and rapid fatigue during exercise was revealed. After transplantation, there was a slight decrease in anxiety (p = 0.91) and a signifi cant decrease in depression (p = 0.011).Conclusion. The expected dynamics was established: in patients with clinical improvement, there was an improvement in the quality of life for most indicators; in patients with stabilization, most quality of life indicators were similar before and after HDIT–AHSCT; with clinical deterioration after HDIT–AHSCT, quality of life parameters worsened. These data indicate the expediency of further optimization of patient selection criteria and various stages of the HDIT–AHSCT in order to obtain optimal clinical results and, accordingly, improve the quality of life of patients.
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Russian Neurological Journal
Russian Neurological Journal Medicine-Neurology (clinical)
CiteScore
0.40
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49
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